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Medical Book from C.H.I.P.S.

Gaucher Disease
edited by Anthony H. Futerman

Gaucher Disease provides valuable information for academic and industry scientists, and clinicians.

Features:

  • Presents not just the most current treatment modalities of this genetic disorder, but the direction of new research in the field
  • Touches on all aspects of the disease including biochemical pathology, diagnosis, treatment, patient management, and ethics
  • Serves as the only fully comprehensive volume on the disease and the first update on technological advances since 1997
  • Contains contributions from world-renowned experts with chapters written by patient organizations, pharmaceutical companies, physicians, public health leaders, and ethicists

Contents

  1. Gaucher Disease: Molecular Biology and Genotype-Phenotype Correlations
  2. Cell Biology and Biochemistry of Acid b-Glucosidase: The Gaucher Disease Enzyme
  3. Saposin C and Other Sphingolipid Activator Proteins, S. Locatelli Hoops
  4. The X-Ray Structure of Human Acid-b-Glucosidase: Implications for Second-Generation Enzyme replacement therapy
  5. Cellular Pathology in Gaucher Disease
  6. The Biochemistry and Cellular Biology of Sphingolipids and Glucosylceramide
  7. The Development of Enzyme replacement therapy for Lysosomal Diseases: Gaucher Disease and Beyond
  8. Gaucher Disease Animal Models
  9. Type 1 Gaucher Disease - Clinical Features
  10. Neuronopathic Gaucher Disease
  11. Pathologic Anatomy of Gaucher Disease
  12. Neuropathological Aspects of Gaucher Disease
  13. Diagnosis and Laboratory Features
  14. Imaging in Gaucher Disease, Focused on Bone Pathology
  15. Radionuclide Evaluation of Gaucher Disease
  16. Epidemiology and Screening Policy
  17. Enzyme replacement therapy for Type I Gaucher Disease
  18. Substrate Reduction Therapy
  19. Pharmacologic Chaperone Therapy for Lysosomal Diseases
  20. The Significance of the Blood-Brain Barrier for Gaucher Disease and Other Lysosomal Storage Diseases
  21. Hematopoietic Stem Cell Transplantation, Stem Cells, and Gene Therapy
  22. Ethical Concerns in Treating Rare Diseases with Expensive Therapy
  23. Societal Aspects in Treating Rare Diseases with Expensive Therapy
  24. Gaucher Disease as a Model for an Orphan Disease: Medical Aspects
  25. Meeting the Needs of Patients with Gaucher Disease: Pioneering a Sustainable Model for Ultra-Orphan Diseases
  26. Patients' Perspective
  27. Societal Perspective: Comment
  28. Gaucher Associations Around the World

Index

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Gaucher Disease
edited by Anthony H. Futerman
2006 544 pages $178.95 + shipping
Texas residents please add 6.75 % sales tax

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